MHRA-102342-PIP01-26

Key Facts

Active Substance
Active Substance:
  • Sepiapterin
Invented Name
  • Sephience
  • Sephience
  • Sephience
  • Sephience
  • Sephience
  • Sephience
  • Sephience
  • Sephience
PIP Number MHRA-102342-PIP01-26
Pharmaceutical form(s)
Pharmaceutical form(s):
  • ORAL POWDER
Therapeutic area
Therapeutic area:
  • Endocrinology-Gynaecology-Fertility-metabolism
Conditions / Indications
Conditions / Indications:
  • Treatment of hyperphenylalaninaemia
Route(s) of administration
Route(s) of administration:
  • Oral use
PIP applicant
  • PTC Therapeutics International Limited
  • Country Ireland
  • Tel 00443450754864
  • Email medinfo@ptcbio.com
Decision Type
Decision Type
P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s)
Compliance Check
Compliance Check
Yes
Compliance Check Decision Date
Compliance Check Procedure Number
Compliance procedure number
MHRA-102342-PIP01-26-C1

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):Sepiapterin.pdf
Published Date 04/09/2026